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EnglishWe characterized disease progression over 48 weeks among boys receiving deflazacort versus prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials. Ambulatory boys with DMD receiving placebo in the Phase 3 ataluren (n=115) and tadalafil (n=116) trials were included. The trials required ≥ 6 months of prior corticosteroid use and stable baseline dosing. Associations between corticosteroid and 48-week changes in ambulatory function were estimated using mixed models. Adjusted differences between corticosteroid groups were pooled in a meta-analysis. In the meta-analysis, deflazacort-treated patients versus prednisone/prednisolone-treated patients experienced, on average, lower declines of 28.3 meters on six-minute walk distance [95% confidence interval: (5.7, 50.9)], 2.9 seconds on rise from supine, [(0.9, 4.9)], 2.3 seconds on 4 stair climb [(0.5, 4.1)], and 2.9 points [(0. 1, 5.8)] on the North Star Ambulatory Assessment linearized score. Deflazacort-treated patients experienced significantly lower functional decline over 48 weeks.
| Titolo: | Deflazacort versus prednisone treatment for Duchenne muscular dystrophy: a meta-analysis of disease progression rates in recent multicenter clinical trials | |
| Autori: | ||
| Data di pubblicazione: | 2020 | |
| Rivista: | ||
| Abstract: | We characterized disease progression over 48 weeks among boys receiving deflazacort versus prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials. Ambulatory boys with DMD receiving placebo in the Phase 3 ataluren (n=115) and tadalafil (n=116) trials were included. The trials required ≥ 6 months of prior corticosteroid use and stable baseline dosing. Associations between corticosteroid and 48-week changes in ambulatory function were estimated using mixed models. Adjusted differences between corticosteroid groups were pooled in a meta-analysis. In the meta-analysis, deflazacort-treated patients versus prednisone/prednisolone-treated patients experienced, on average, lower declines of 28.3 meters on six-minute walk distance [95% confidence interval: (5.7, 50.9)], 2.9 seconds on rise from supine, [(0.9, 4.9)], 2.3 seconds on 4 stair climb [(0.5, 4.1)], and 2.9 points [(0. 1, 5.8)] on the North Star Ambulatory Assessment linearized score. Deflazacort-treated patients experienced significantly lower functional decline over 48 weeks. | |
| Handle: | http://hdl.handle.net/11570/3145121 | |
| Appare nelle tipologie: | 14.a.1 Articolo su rivista |
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